EFFICACY AND SAFETY OF IPTACOPAN IN PARTICIPANTS WITH C3 GLOMERULOPATHY: C3G EXTENSION TRIAL INTERIM RESULTS FROM THE PHASE 3 APPEAR-C3G PARTICIPANTS

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Abstract Title *

EFFICACY AND SAFETY OF IPTACOPAN IN PARTICIPANTS WITH C3 GLOMERULOPATHY: C3G EXTENSION TRIAL INTERIM RESULTS FROM THE PHASE 3 APPEAR-C3G PARTICIPANTS

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Co-author 1

Carla M. Nester cnester@uiowa.edu Stead Family Children’s Hospital University of Iowa Iowa United States *

Co-author 2

Richard J.H. Smith richard-smith@uiowa.edu Carver College of Medicine University of Iowa Iowa United States -

Co-author 3

David Kavanagh david.kavanagh@newcastle.ac.uk Translational and Clinical Research Institute Newcastle University Newcastle upon Tyne United Kingdom - National Renal Complement Therapeutics Centre Royal Victoria Infirmary Newcastle upon Tyne United Kingdom

Co-author 4

Marina Vivarelli marina.vivarelli@opbg.net Bambino Gesù Children's Hospital IRCCS Rome Italy -

Co-author 5

Giuseppe Remuzzi giuseppe.remuzzi@marionegri.it Istituto di Ricerche Farmacologiche Mario Negri IRCCS Bergamo Italy -

Co-author 6

Ming-Hui Zhao mhzhao@bjmu.edu.cn Peking University First Hospital Beijing Beijing China -

Co-author 7

Edwin K.S. Wong Edwin.wong1@nhs.net National Renal Complement Therapeutics Centre Royal Victoria Infirmary Newcastle upon Tyne United Kingdom -

Co-author 8

Yaqin Wang yaqin.wang@novartis.com Novartis Pharmaceuticals Corporation East Hanover New Jersey United States -

Co-author 9

Rubeen K. Israni rubeen.israni@novartis.com Novartis Pharmaceuticals Corporation East Hanover New Jersey United States -

Co-author 10

Hari V. Kalluri hari.kalluri@novartis.com Novartis Pharmaceuticals Corporation East Hanover New Jersey United States -

Co-author 11

Induja Krishnan induja.krishnan@novartis.com Novartis Pharmaceuticals Corporation East Hanover New Jersey United States -

Co-author 12

Jean Grisouard jean-1.grisouard@novartis.com Novartis Pharma AG Basel Basel Switzerland -

Co-author 13

Andrew S. Bomback asb68@cumc.columbia.edu David Kock Jr Glomerular Kidney Center Columbia University Irving Medical Center New York United States -

Co-author 14

Co-author 15

Introduction

The Phase 3 APPEAR-C3G study evaluated the efficacy, safety, and tolerability of iptacopan vs placebo in participants with C3G

Methods

APPEAR-C3G (NCT04817618) was a multicenter, randomized, double-blind, placebo-controlled, pivotal Phase 3 study that included adult participants with biopsy-confirmed C3 glomerulopathy (C3G). Upon completion of the study treatment at 12 months in APPEAR-C3G, participants had the option to continue open-label iptacopan treatment in the C3G extension study (NCT03955445). Here we present the results from the extension study with iptacopan treatment up to 24 months

Results

Of 73 participants completing the 12-month treatment, 66 participants entered the extension study. In the iptacopan arm, the geometric mean reduction in first morning void (FMV) urine protein-creatinine ratio (UPCR) at Month 12 (35.9%), was sustained up to Month 24 (34.2%) compared to baseline (Figure). In the placebo-iptacopan switch arm, the reduction at Month 12 (34.0%) was also sustained up to Month 24 (29.7%). The mean pre-treatment annualized estimated glomerular filtration rate (eGFR) slope (95% CI) in all participants (n=74) showed rapid decline with −7.22 mL/min/1.73m2/year (−10.10, −4.35). After initiation of iptacopan treatment, there was stabilization of annualized eGFR slope (95% CI) at −0.29 mL/min/1.73m2/year (−3.88, 3.31) assessed over 24 months. Iptacopan demonstrated a favorable safety profile with no new safety signals identified

Conclusion

Iptacopan demonstrated sustained reduction in proteinuria and stabilization of eGFR slope with treatment up to 24 months among APPEAR-C3G participants who rolled over to the long-term extension study. Iptacopan was well tolerated with a favorable safety profile in participants with C3G

This abstract was also submitted to the American Society of Nephrology (ASN) Kidney Week 2025 Congress, November 6–9, in Houston, TX, USA. Encore submission of this abstract to WCN 2026 is permitted by the organizers of the original meeting (ASN 2025).

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